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N Engl J Med. 2014 Aug 21;371(8):699-710. doi: 10.1056/NEJMoa1401731.

Controlled trial of transfusions for silent cerebral infarcts in sickle cell anemia.

Author information

1
From the Department of Pediatrics, Division of Hematology-Oncology, Vanderbilt-Meharry Center of Excellence in Sickle Cell Disease, Vanderbilt University School of Medicine, Nashville (M.R.D.); Department of Ophthalmology and Visual Sciences, Division of Biostatistics (M.G.), Departments of Radiology and Pediatrics (R.C.M.), Neurology and Pediatrics (M.J.N.), and Psychology (D.A.W.), the Program in Occupational Therapy and Department of Pediatrics Hematology-Oncology (A.A.K.), and the Division of Biostatistics and Department of Internal Medicine (J.P.M.), Washington University School of Medicine, St. Louis; Department of Pediatrics, Division of Hematology-Oncology, Wayne State University, Detroit (S.A.S.); Center for Cancer and Blood Disorders, Children's National Medical Center, Department of Pediatrics, George Washington University Medical Center (E.R.M.), and Department of Pediatrics, Division of Hematology-Oncology, Georgetown University Hospital (C.E.G.) - all in Washington, DC; Department of Pediatrics, Division of Hematology-Oncology, University of Alabama at Birmingham, Birmingham (T.H.H.); Department of Pediatrics, Division of Hematology-Oncology, University of Mississippi Medical Center, Jackson (S.M.); Department of Paediatrics, Evelina Children's Hospital, St. Thomas' Hospital NHS Trust (B.P.D.I.), Department of Pediatric Hematology, Royal London Hospital, Barts Health NHS Trust (P.T.T.), and the Neurosciences Unit, Institute of Child Health, University College London (F.J.K.) - all in London; Hospital for Sick Children, Department of Paediatrics, Haematology-Oncology, University of Toronto, Toronto (M.K.-A.); Division of Hematology-Oncology, Department of Pediatrics, UT Southwestern Medical Center, Dallas (T.L.M.C.); Département Pédiatrie, Hôpital Intercommunal de Creteil, Creteil, France (A.K.); Department of Pediatrics, Division of Hematology-Oncology, Baylor College of Medicine, Houston (G.A.); Department of Pediatrics, Hematology-Oncology, Univer

Abstract

BACKGROUND:

Silent cerebral infarcts are the most common neurologic injury in children with sickle cell anemia and are associated with the recurrence of an infarct (stroke or silent cerebral infarct). We tested the hypothesis that the incidence of the recurrence of an infarct would be lower among children who underwent regular blood-transfusion therapy than among those who received standard care.

METHODS:

In this randomized, single-blind clinical trial, we randomly assigned children with sickle cell anemia to receive regular blood transfusions (transfusion group) or standard care (observation group). Participants were between 5 and 15 years of age, with no history of stroke and with one or more silent cerebral infarcts on magnetic resonance imaging and a neurologic examination showing no abnormalities corresponding to these lesions. The primary end point was the recurrence of an infarct, defined as a stroke or a new or enlarged silent cerebral infarct.

RESULTS:

A total of 196 children (mean age, 10 years) were randomly assigned to the observation or transfusion group and were followed for a median of 3 years. In the transfusion group, 6 of 99 children (6%) had an end-point event (1 had a stroke, and 5 had new or enlarged silent cerebral infarcts). In the observation group, 14 of 97 children (14%) had an end-point event (7 had strokes, and 7 had new or enlarged silent cerebral infarcts). The incidence of the primary end point in the transfusion and observation groups was 2.0 and 4.8 events, respectively, per 100 years at risk, corresponding to an incidence rate ratio of 0.41 (95% confidence interval, 0.12 to 0.99; P=0.04).

CONCLUSIONS:

Regular blood-transfusion therapy significantly reduced the incidence of the recurrence of cerebral infarct in children with sickle cell anemia. (Funded by the National Institute of Neurological Disorders and Stroke and others; Silent Cerebral Infarct Multi-Center Clinical Trial ClinicalTrials.gov number, NCT00072761, and Current Controlled Trials number, ISRCTN52713285.).

PMID:
25140956
PMCID:
PMC4195437
DOI:
10.1056/NEJMoa1401731
[Indexed for MEDLINE]
Free PMC Article

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